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Journal Articles

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Wolfgang Miesbach, Karina Meijer, Michiel Coppens, Peter Kampmann, Robert Klamroth, Roger Schutgens, Marco Tangelder, Giancarlo Castaman, Joachim Schwäble, Halvard Bonig, Erhard Seifried, Federica Cattaneo, Christian Meyer, Frank W. G. Leebeek

Blood (2018) 131 (9): 1022–1031.

https://doi.org/10.1182/blood-2017-09-804419

Published: 2018

Includes: Supplemental data

Journal Articles

Novel Human Factor VIII Variant with Impaired Intracellular Processing Exhibits Enhanced In Vivo Efficacy

Giang N. Nguyen, B.S., Christian M. Wood, Tanner Dirstine, B.S., Robert J. Davidson, B.S., Denise E. Sabatino, PhD

Blood (2016) 128 (22): 256.

https://doi.org/10.1182/blood.V128.22.256.256

Published: 2016

Journal Articles

Beta-Globin Gene Targeting in Human Hematopoietic Stem Cells Using Cas9 Ribonucleoprotein and rAAV6

Daniel Patrick Dever, PhD, Matthew Porteus, MD PhD

Blood (2016) 128 (22): 2310.

https://doi.org/10.1182/blood.V128.22.2310.2310

Published: 2016

Journal Articles

Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic Factor VIII Levels in Non-Human Primates

Xavier M. Anguela, PhD, Liron Elkouby, PhD, Raffaella Toso, PhD, Marti DiPietro, MS, Robert J. Davidson, Katherine A. High, MD, Denise E. Sabatino, PhD

Blood (2015) 126 (23): 199.

https://doi.org/10.1182/blood.V126.23.199.199

Published: 2015

Journal Articles

Bioengineered Coagulation Factor VIII Enables Long-Term Correction Of Murine Hemophilia A Following Liver-Directed Adeno-Associated Viral Vector Delivery

Harrison C. Brown, BBA, Jordan E Shields, Shangzhen Zhou, MD, J. Fraser Wright, PhD, H. Trent Spencer, PhD, Christopher B Doering, PhD

Blood (2013) 122 (21): 4210.

https://doi.org/10.1182/blood.V122.21.4210.4210

Published: 2013

Journal Articles

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

Blood (2011) 118 (21): 5.

https://doi.org/10.1182/blood.V118.21.5.5

Published: 2011

Journal Articles

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

Amit Nathwani, MD, PhD, Edward Tuddenham, MD, Cecilia Rosales, Jenny McIntosh, Anne Riddell, Pradip Rustagi, Bertil Glader, Mark Kay, MD, PhD, James Allay, PhD, John Coleman, Susan Sleep, Katherine A. High, MD, Federico Mingozzi, PhD., John T. Gray, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff

Blood (2010) 116 (21): 248.

https://doi.org/10.1182/blood.V116.21.248.248

Published: 2010

Journal Articles

Gene Therapy of Chronic Myelogenous Leukemia Using Pseudotyped Recombinant Adeno-Associated Viral Vectors.

Leopold Sellner, MD, Marlon R Veldwijk, PhD, Jürgen A. Kleinschmidt, PhD, Stephanie Laufs, PhD, Julian Topaly, MD, W. Jens Zeller, MD, Frederik Wenz, MD, Stefan Fruehauf, MD

Blood (2009) 114 (22): 4506.

https://doi.org/10.1182/blood.V114.22.4506.4506

Published: 2009

Journal Articles

Efficient Gene Transfer into Human CD34 + Peripheral Blood Progenitor Cells Using Pseudotyped Recombinant Adeno-Associated Viral Vectors

Leopold Sellner, Marlon Veldwijk, PhD, Marius Stiefelhagen, Jurgen A. Kleinschmidt, PhD, Stephanie Laufs, PhD, Julian Topaly, MD, W. Jens Zeller, MD, Frederik Wenz, MD, Stefan Fruehauf, MD

Blood (2008) 112 (11): 4627.

https://doi.org/10.1182/blood.V112.11.4627.4627

Published: 2008

Journal Articles

Directed Evolution of Immune-Escaping Adeno-Associated Virus Vectors

Stephan Maersch, Anke Huber, Michael Hallek, M.D., Hildegard Buening, Luca Perabo, PhD

Blood (2008) 112 (11): 3532.

https://doi.org/10.1182/blood.V112.11.3532.3532

Published: 2008

Journal Articles

Safety of Recombinant Adeno-Associated Viral Vectors in a Large Animal Model.

Patricia Favaro, PhD, Harre D. Downey, Federico Mingozzi, PhD, Fraser Wright, PhD, Bernd Hauck, PhD, Katherine A. High, MD, Valder R. Arruda, MD, PhD

Blood (2007) 110 (11): 2586.

https://doi.org/10.1182/blood.V110.11.2586.2586

Published: 2007

Journal Articles

Novel Efficient Primary Human Peripheral Blood Progenitor Cell-Targeted Recombinant Adeno-Associated Viral Vectors.

Marlon R. Veldwijk, Ph.D., Leopold Sellner, Marius Stiefelhagen, Jürgen A. Kleinschmidt, Ph.D., Stephanie Laufs, Ph.D., Julian Topaly, M.D., Frederik Wenz, M.D., W. Jens Zeller, M.D., Stefan Fruehauf, MD

Blood (2007) 110 (11): 5144.

https://doi.org/10.1182/blood.V110.11.5144.5144

Published: 2007

Journal Articles

A Novel Human Factor IX Expression Cassette Packaged as Self-Complementary DNA Dimers in Adeno-Associated Virus Vectors Results in Dramatically Improved Liver Transduction, Significantly Improving Prospects for Hemophilia B Gene Therapy.

John T. Gray, PhD, Andrew M. Davidoff, MD, Amit C. Nathwani, MD, PhD

Blood (2004) 104 (11): 3181.

https://doi.org/10.1182/blood.V104.11.3181.3181

Published: 2004

Journal Articles

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

Andrew M. Davidoff, Catherine Y. C. Ng, Junfang Zhou, Yunyu Spence, Amit C. Nathwani

Blood (2003) 102 (2): 480–488.

https://doi.org/10.1182/blood-2002-09-2889

Published: 2003

Journal Articles

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA

Amit C. Nathwani, Andrew Davidoff, Hideki Hanawa, Jun-Fang Zhou, Elio F. Vanin, Arthur W. Nienhuis

Blood (2001) 97 (5): 1258–1265.

https://doi.org/10.1182/blood.V97.5.1258

Published: 2001

Journal Articles

Adeno-Associated Virus Vectors and Hematology

David W. Russell, Mark A. Kay

Blood (1999) 94 (3): 864–874.

https://doi.org/10.1182/blood.V94.3.864.415k34_864_874

Published: 1999

Journal Articles

Integration of adeno-associated virus vectors in CD34+ human hematopoietic progenitor cells after transduction

G Fisher-Adams, KK Jr Wong, G Podsakoff, SJ Forman, S Chatterjee

Blood (1996) 88 (2): 492–504.

https://doi.org/10.1182/blood.V88.2.492.bloodjournal882492

Published: 1996

NARROW

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Novel Human Factor VIII Variant with Impaired Intracellular Processing Exhibits Enhanced In Vivo Efficacy

Beta-Globin Gene Targeting in Human Hematopoietic Stem Cells Using Cas9 Ribonucleoprotein and rAAV6

Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic Factor VIII Levels in Non-Human Primates

Bioengineered Coagulation Factor VIII Enables Long-Term Correction Of Murine Hemophilia A Following Liver-Directed Adeno-Associated Viral Vector Delivery

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

Gene Therapy of Chronic Myelogenous Leukemia Using Pseudotyped Recombinant Adeno-Associated Viral Vectors.

Efficient Gene Transfer into Human CD34 + Peripheral Blood Progenitor Cells Using Pseudotyped Recombinant Adeno-Associated Viral Vectors

Directed Evolution of Immune-Escaping Adeno-Associated Virus Vectors

Safety of Recombinant Adeno-Associated Viral Vectors in a Large Animal Model.

Novel Efficient Primary Human Peripheral Blood Progenitor Cell-Targeted Recombinant Adeno-Associated Viral Vectors.

A Novel Human Factor IX Expression Cassette Packaged as Self-Complementary DNA Dimers in Adeno-Associated Virus Vectors Results in Dramatically Improved Liver Transduction, Significantly Improving Prospects for Hemophilia B Gene Therapy.

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA

Adeno-Associated Virus Vectors and Hematology

Integration of adeno-associated virus vectors in CD34+ human hematopoietic progenitor cells after transduction

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American Society of Hematology

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American Society of Hematology

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